Crizanlizumab is a potent and selective IgG2 (immunoglobulin G2) kappa humanized monoclonal antibody that binds to human P-selectin with high affinity, blocking its interaction with its ligands, including P-selectin glycoprotein ligand 1 (PSGL-1). Extensive pre-clinical data have established P-selectin as a key mediator of VOC in SCD (Matsui et al 2001) and suggest that blockade of P-selectin could eliminate or reduce VOC.
The objective of this study is to assess the efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea (HU)/hydroxycarbamide (HC), on VOC rate in Sickle Cell Disease (SCD) patients aged 12 years and older who experience frequent vaso-occlusive crises (VOCs).
SPARKLE study, is a new phase III study developed to confirm the efficacy of crizanlizumab demonstrated in the SUSTAIN study and other open label studies.
Expected impact: Crizanlizumab as an effective treatment option for SCD, with reduction in frequency of VOC, and improvement in patients’ quality of life and life expectancy.
